NCT03634007

Gene Therapy for APOE4 Homozygote of Alzheimer's Disease

A 52-Week, Multicenter, Phase 1/2 Open-label Study to Evaluate the Safety of LX1001 in Participants With APOE4 Homozygote Alzheimer's Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Lexeo Therapeutics · Industry
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

Summary

This clinical trial is an open label, dose-ranging study designed to evaluate gene therapy to treat patients who are APOE4 homozygotes with clinical diagnosis varying from mild cognitive impairment due to Alzheimer's, mild dementia due to Alzheimer's disease, and moderate dementia due to Alzheimer's disease.

Detailed description

The study will assess the safety and toxicity of intrathecal administration of AAVrh.10hAPOE2 (LX1001), serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the complementary deoxyribonucleic acid (cDNA) coding for human apolipoprotein E2 (APOE2), directly to the central nervous system (CNS)/ CSF of APOE4 homozygotes with Alzheimer's disease. All subjects will have evidence of cerebrospinal fluid (CSF) biomarkers consistent with Alzheimer's disease. The study will establish a maximum tolerable dose and generate preliminary evidence regarding whether direct administration of LX1001 to the CNS of those Alzheimer's patients will lead to conversion of the APOE protein isoforms in the CSF of APOE4 homozygotes from APOE4 to APOE2-APOE4.

Conditions

• Alzheimer Disease
• Early Onset Alzheimer Disease

Interventions

Type	Name	Description
BIOLOGICAL	LX1001	LX1001 is a serotype rh.10 AAV gene transfer vector expressing the cDNA coding for human APOE2.

Timeline

• Start date: 2019-11-06
• Primary completion: 2024-11-07
• Completion: 2024-11-07
• First posted: 2018-08-16
• Last updated: 2025-10-20

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study